Navigation Links
Regulus Presents Positive Preclinical Data Demonstrating that microRNA-21 Plays an Important Role in Alport Syndrome

LA JOLLA, Calif., Nov. 8, 2013 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced that positive new preclinical data demonstrating that microRNA-21 (miR-21) plays an important role in Alport Syndrome will be presented at the American Society of Nephrology's Kidney Week 2013 meeting being held November 5-10 in Atlanta, GA.

In a poster presentation titled "Identification of the Pathologic Role of miR-21 in Alport's Kidney Disease" on Friday, November 8, 2013 from 10:00 a.m. EST to 12:00 p.m. EST, Regulus and Genzyme, a Sanofi company, presented new preclinical data demonstrating that miR-21 plays an important role in the disease progression of Alport Syndrome in collagen 4A3 deficient mice.  The study assessed urine albumin, kidney function, pathology and gene expression in the mice at 4, 6, 9, 12 and 15 weeks of age.  Efficacy of an anti-miR-21 candidate was assessed in mice starting at 5 weeks of age.  Urine albumin, renal function, kidney pathology and lifespan were used as efficacy endpoints.  Treatment with an anti-miR-21 candidate significantly improved renal function, significantly reversed regulated genes and pathways associated with renal pathology, and increased the lifespan of the mice by 20 percent. The poster can be accessed on Regulus' website,

"We are extremely pleased with the results from this study and believe these positive data validate that treatment with an anti-miR-21 candidate may be an important new therapy for patients with Alport Syndrome," said Neil W. Gibson, Ph.D., Chief Scientific Officer at Regulus. "These data bring us one step closer toward the selection of an anti-miR-21 clinical development candidate and we remain excited about the potential to bring this innovative treatment to patients with this devastating disease." 

Additionally, microRNA therapeutics will be discussed in two oral presentations at the meeting:

  • On Friday, November 8, 2013 at 2:00 p.m. EST, Jeremy Duffield M.D., Ph.D., Regulus' collaborator at the University of Washington, will give an oral presentation titled "MicroRNAs Are Novel Therapeutic Targets to Treat Kidney Injury and Fibrosis"; and  
  • On Saturday, November 9, 2013 at 5:42 p.m. EST, Regulus scientists and representatives from its collaborator at the University of Washington will give an oral presentation titled "Anti-miR21 Protects Collagen 4A3 Deficient Mice from Progression of Alport Disease by Decreasing Oxidative Stress"

About miR-21 and Alport Syndrome

According to the National Institutes of Health, Alport Syndrome occurs in approximately 1 in 50,000 newborns.  Alport syndrome is a genetic condition characterized by kidney disease, hearing loss, and eye abnormalities.  The kidneys become less able to function as this condition progresses, resulting in end-stage renal disease (ESRD).

Mutations in the COL4A3, COL4A4, and COL4A5 genes cause Alport Syndrome. These genes provide instructions for making one component of a protein called type IV collagen. This protein plays an important role in the kidneys, specifically in structures called glomeruli. Glomeruli are clusters of specialized blood vessels that remove water and waste products from blood and create urine. Mutations in these genes result in abnormalities of the type IV collagen in glomeruli, which prevents the kidneys from properly filtering the blood and allows blood and protein to pass into the urine.

miR-21 is a 22-mer non-coding RNA that negatively regulates gene/networks and has been reported to be up-regulated in fibrotic kidney diseases in both animal models and human patients (Chau, B. N. et al. Sci Transl Med. 2012; Zhong X, et al. (2013) Diabetologia (2013).  Previous reports have demonstrated that treatment with an anti-miR-21 significantly attenuates chronic kidney disease progression in a collagen 4A3 deficient mouse model.  Regulus' miR-21 therapeutic development program, which is partnered with Sanofi, is currently in preclinical testing for both kidney fibrosis and hepatocellular carcinoma.

About Regulus

Regulus Therapeutics Inc. (NASDAQ: RGLS) is a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs. Regulus is uniquely positioned to leverage a mature therapeutic platform that harnesses the oligonucleotide drug discovery and development expertise of Alnylam Pharmaceuticals, Inc. and Isis Pharmaceuticals, Inc., which founded the company.   Regulus has a well-balanced microRNA therapeutic pipeline entering clinical development, an emerging microRNA biomarkers platform to support its therapeutic programs, and a rich intellectual property estate to retain its leadership in the microRNA field.  Regulus intends to focus its proprietary efforts on developing microRNA therapeutics for oncology indications and orphan diseases and is currently advancing several programs toward clinical development in oncology, fibrosis and metabolic diseases.  Regulus is also developing RG-101, a GalNAc-conjugated anti-miR targeting microRNA-122, for the treatment of chronic hepatitis C virus infection.  Regulus' commitment to innovation and its leadership in the microRNA field have enabled the formation of strategic alliances with AstraZeneca, GlaxoSmithKline and Sanofi.  In addition, Regulus has formed a research collaboration with Biogen Idec around its emerging microRNA biomarkers platform. 

For more information, please visit

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with Regulus' expectations regarding future therapeutic and commercial potential of Regulus' business plans, technologies and intellectual property related to microRNA therapeutics being discovered and developed by Regulus.  Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "intends," "will," "goal," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Regulus' current expectations and involve assumptions that may never materialize or may prove to be incorrect.  Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs.  These and other risks concerning Regulus' programs are described in additional detail in Regulus' SEC filings.  All forward-looking statements contained in this press release speak only as of the date on which they were made. Regulus undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

SOURCE Regulus Therapeutics Inc.
Copyright©2012 PR Newswire.
All rights reserved

Related medicine technology :

1. Regulus Announces Timing for Third Quarter 2013 Financial Results Webcast and Conference Call
2. Regulus Announces Significant Progress in Strategic Alliance with AstraZeneca and Selection of a New Pre-Clinically Validated microRNA Oncology Target
3. Regulus Reports Second Quarter 2013 Financial Results and Recent Highlights
4. Regulus Announces Timing for Second Quarter 2013 Financial Results Webcast and Conference Call
5. Regulus Names Stelios Papadopoulos, Ph.D. as Chairman of its Board of Directors
6. Regulus to Present at Deutsche Bank 38th Annual Healthcare Conference
7. Regulus Provides Update on Road to the Clinic Strategy and Reports First Quarter 2013 Financial Results and Recent Highlights
8. Regulus Announces Timing for First Quarter 2013 Financial Results Webcast and Conference Call
9. Regulus Appoints William H. Rastetter, Ph.D. to its Board of Directors
10. Regulus to Present at Future Leaders in the Biotech Industry Conference
11. Regulus to Present at Cowen and Company 33rd Annual Healthcare Conference
Post Your Comments:
(Date:11/30/2015)... Next week, December 2-3, BIOMEDevice San ... co-located events covering the latest in Medtech innovation, Wearable ... draw more than 3,000 design industry professionals to the ... events, combined show floor will host more than 300 ... --> --> BIOMEDevice features suppliers in ...
(Date:11/30/2015)...  IBA Molecular North America, Inc. (IBAMNA), a U.S. ... that as of January 1, 2016, it will do ... to rebrand the company reflects a refined vision for ... close relationship with Zevacor Molecular.  Both IBAMNA and Zevacor ... Peter Burke , Vice President Sales ...
(Date:11/30/2015)... , Nov. 30, 2015 Nautilus Medical Inc. ... Image Management platform ( ). The release of ... from RSNA 2015 (Radiology Society North America) in ... in the U.S. --> ... that enables access to radiology studies worldwide via a ...
Breaking Medicine Technology:
(Date:11/30/2015)... ... , ... According to Los Angeles bariatric surgeon Michael Feiz, M.D., F.A.C.S., many ... real hunger, but instead by a hormone called ghrelin which (often prematurely) signals ... are aware that weight loss surgery can help patients lose weight by restricting the ...
(Date:11/30/2015)... Miami, FL (PRWEB) , ... November 30, 2015 ... ... added a comparison chart and ingredient list of its hemorrhoid ointment to its ... “fast, effective pain relief for people suffering from hemorrhoids. Adding the comparison chart ...
(Date:11/30/2015)... ... November 30, 2015 , ... Using a combination of two blood sugar tests ... adults, according to a new study by researchers at the School of Public Health ... Adults: Using Combinations of Blood Glucose Tests ,” published in Frontiers in Public Health, ...
(Date:11/30/2015)... ... November 30, 2015 , ... The recently ... Poison Data System (NPDS) reveals that in 2014, someone called a poison center ... two million of which were human exposure cases. , The American Association of ...
(Date:11/30/2015)... Reading, PA (PRWEB) , ... November 30, 2015 ... ... Ashland Specialty Ingredients (ASI) as their exclusive channel partner for the Nutraceutical Specialties ... supplements markets in the US, effective immediately. , “We are pleased to ...
Breaking Medicine News(10 mins):